On August 11, 2021 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the second quarter ended June 30, 2021 (Press release, Tracon Pharmaceuticals, AUG 11, 2021, View Source [SID1234586371]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

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"We remain on track to announce interim efficacy data from the pivotal ENVASARC trial by the end of this year and final data in 2022," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Last week the IDMC recommended the study proceed as planned following review of interim safety data and we will continue to focus our efforts on enrolling this pivotal trial. Following our recent public offering, our balance sheet provides us with cash runway into 2023."

Recent Corporate Highlights

Envafolimab

In June, we received orphan drug designation for envafolimab in soft tissue sarcoma based on clinical data demonstrating confirmed objective partial responses by RECIST with duration of response in excess of six months, in two of five patients with refractory metastatic alveolar soft part sarcoma (ASPS) who received single agent envafolimab in Phase 1 clinical trials conducted by our partners, 3D Medicines and Alphamab Oncology.

On August 6, the Independent Data Monitoring Committee (IDMC) recommended the ENVASARC trial proceed as planned following the review of more than three months of safety data from more than 20 patients enrolled in the trial as of May.

In June, we presented the study design of the pivotal Phase 2 ENVASARC trial of single agent envafolimab and envafolimab given with Yervoy (ipilimumab) at the 2021 ASCO (Free ASCO Whitepaper) virtual annual meeting.
Corporate

In July, we announced an underwritten public offering for gross proceeds of approximately $15.0 million. We estimate that the proceeds will extend our cash runway into 2023, past expected final top-line data for ENVASARC.

In the second quarter we enhanced our senior management team with multiple new hires, including a Head of Regulatory and Head of Biometrics, both of whom have BLA filing experience.

In July, the book Unnecessary Expense: An Antidote to the Billion Dollar Drug Problem, authored by TRACON senior management to be published by ForbesBooks became available for preorder on Amazon. Publication is expected in September.
Other

In June, we presented preliminary Phase 1 data for the CD73 antibody uliledlimab (TJ4309) at the 2021 ASCO (Free ASCO Whitepaper) virtual annual meeting.
Expected Key Upcoming Milestones

Interim ENVASARC efficacy data by end of 2021.

Request FDA breakthrough therapy designation or fast track designation for envafolimab by end of 2021, assuming positive interim efficacy data.

Decision on the envafolimab New Drug Application in MSI-H/dMMR cancer that is under priority review by the Chinese National Medical Products Administration.
Second Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments were $25.6 million at June 30, 2021, compared to $36.1 million at December 31, 2020. The cash balance at June 30, 2021, does not include the proceeds from the $15 million underwritten public offering in July, which extends the Company’s cash runway into 2023.

Research and development expenses for the second quarter of 2021 were $3.1 million, compared to $2.2 million for the second quarter of 2020.

General and administrative expenses for the second quarter of 2021 were $6.1 million, compared to $2.1 million for the second quarter of 2020. The increase was primarily attributable to legal expenses incurred with the now stayed lawsuit filed by I-Mab in the Delaware Court of Chancery and ongoing arbitration on the TJ4309 and bispecific agreements. We expect the second quarter of 2021 to be the high point for general and administrative expenses this year.

Net loss for the second quarter of 2021 was $8.9 million, compared to $4.5 million for the second quarter of 2020.
Conference Call Details

Wednesday, August 11, at 4:30 PM Eastern Time / 1:30 PM Pacific Time
Domestic: 855-779-9066
International: 631-485-4859
Conference ID: 3067769
A live webcast of the conference call will be available online from the Investor/Events and Presentations page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, has been studied in a completed Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients in China and is being studied in an ongoing Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China, with both Chinese trials sponsored by 3D Medicines. TRACON’s partners Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of the KEYNOTE-164 clinical trial.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled into cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.

About TRC102

TRC102 (methoxyamine) is a novel, small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the U.S. FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On August 11, 2021 Advaxis, Inc. (Nasdaq: ADXS) ("Advaxis" or the "Company"), a clinical-stage biotechnology company focused on the development and commercialization of proprietary Lm-based antigen delivery products, reported that it has received a letter indicating that following the Company’s hearing before the Nasdaq Hearings Panel (the "Panel"), the Panel determined to grant the Company an extension through November 22, 2021, to comply with Nasdaq’s $1.00 Minimum Bid Price Rule and complete its previously announced merger transaction with Biosight, Ltd. ("Biosight") (Press release, Advaxis, AUG 11, 2021, View Source [SID1234586286]). On July 6, 2021, Advaxis announced that it had entered into a merger agreement with Biosight, a privately held, Israel-based pharmaceutical company developing innovative therapeutics for hematological malignancies and disorders.

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Pursuant to the Nasdaq Listing Rules, the combined company will be required to meet all applicable initial listing requirements upon the closing of the merger, including the $4 per share price requirement. While there can be no assurance, the Company believes that it will be able to close the merger and demonstrate compliance with all applicable requirements for initial listing on The Nasdaq Capital Market on or before November 22, 2021.

On August 11, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a preclinical-stage biopharmaceutical company focused on the discovery and development of irreversible small molecules to treat patients with genetically defined cancers, reported financial results for the second quarter of 2021 (Press release, Biomea Fusion, AUG 11, 2021, View Source [SID1234586332]).

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"We continue to make notable progress as we rapidly advance BMF-219 toward a planned entry into the clinic and continue to grow our company and pipeline," said Thomas Butler, Biomea’s CEO and Chairman of the Board. "In the second quarter, we successfully completed IND-enabling studies for BMF-219 and are now in the final stages of completing our IND submission to the FDA to advance BMF-219 to a Phase I study for the treatment of patients with menin-dependent AML and ALL. We also continued to make important progress pursuing our broad, ambitious, preclinical strategy focused on exploring the potential of this novel irreversible small molecule across a number of menin-dependent liquid and solid tumors, including DLBCL."

Mr. Butler continued, "While our primary focus is oncology drug development, there are multiple diseases where the menin pathway is implicated and where an irreversibly binding small molecule approach may have an important impact, including for patients with type 2 diabetes. At our core, we feel a profound responsibility to help patients with our novel chemistry. To that end, we look forward to seeing the results of our exploratory pathway validation in type 2 diabetes next year."

Business Highlights

Completed IND-enabling studies with irreversibly binding menin inhibitor BMF-219. Biomea successfully conducted IND-enabling studies with BMF-219, an irreversible menin inhibitor for the treatment of patients with menin-dependent AML and ALL. Biomea anticipates submitting the IND during the second half of 2021.

Continued preclinical studies of BMF-219 in DLBCL. Biomea continues to explore the potential of BMF-219 for a number of menin-dependent liquid and solid tumor cancers, including a subset of DLBCL, with a number of preclinical studies currently underway. The Company plans to report its findings in DLBCL in the first quarter of 2022. Despite the high dependency of several cancers on menin, Biomea Fusion is not aware of any menin inhibitors currently in clinical development for these cancer types.

Initiated pathway validation studies in diabetes. Based on a growing body of internal and external scientific evidence, Biomea has initiated key preclinical studies to explore the potential of its irreversible menin inhibitors as a treatment for type 2 diabetes. The Company plans to report its findings in the first quarter of 2022. Biomea Fusion is not aware of any menin inhibitors currently in clinical development for diabetes.
Expanded team and in-house research capabilities to support long-term growth and clinical and preclinical development plans. Biomea strengthened its executive team with the appointments of Franco Valle as Chief Financial Officer, Alex Cacovean, M.D. as Executive Medical Director and Sasha Blaug Ph.D. as Senior VP of Corporate Development. Biomea has successfully grown its headcount this year to date by 29 for a total of 41 current team members. The Company has also completed the buildout of its own laboratory facilities to further support the research and preclinical pipeline development of its irreversible platform.
Strengthened the Company’s Board of Directors. In the second quarter Biomea appointed Sumita Ray J.D., Chief Legal and Administrative Officer at Calithera Biosciences, Inc., to its Board. Ms. Ray is an industry veteran with over 20 years of expertise in FDA regulatory law, global health care law and compliance, brand support, product launches, collaborations and alliances.
Financial Highlights

Second Quarter 2021 Year to Date Financial Results

Biomea reported a net loss attributable to common stockholders of $14.3 million for the first six months of 2021, compared to a net loss of $0.7 million for the same period in 2020.

Research and development expenses were $9.0 million for the first six months of 2021, compared to $0.6 million for the same period in 2020. The increase of $8.4 million was primarily due to an increase in personnel-related expenses, as well as an increase in pre-clinical development costs, including manufacturing and external consulting, related to the IND-enabling studies for BMF-219.

General and administrative expenses were $5.3 million for the first six months of 2021, compared to $0.1 million for the same period in 2020. The increase of $5.2 million was primarily due to higher personnel-related expenses and other corporate costs to support the Company’s expanding operations, including legal and accounting.

As of June 30, 2021, the Company had cash, cash equivalents, restricted cash, and investments of $203.0 million.

On August 11, 2021 Immunetune, a preclinical-stage biotech developing next-generation DNA vaccines against cancer and infectious diseases, reported the appointment of Sijme Zeilemaker as Chief Executive Officer (Press release, ImmuneTune, AUG 11, 2021, View Source [SID1234586349]). With his experience in several oncology biotech companies ranging from preclinical to Phase II clinical development and skillset in business development and investor relations, he will be responsible for bringing Immunetune into the next phase of the company.

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"It is great to be joining a biotech working on truly differentiated technologies to bring a potentially best-in-class program into the clinic. Having seen the forefront of innovation in immuno-oncology the last few years, Immunetune has the right approach and results to make a difference. Gerben and his team have done an impressive amount of work and it is such an exciting phase to be joining them," stated Sijme Zeilemaker.

"We are thrilled bringing Sijme on board to strengthen our team at this pivotal point in time. With his experience with both big pharma partners and international life science investors, he knows exactly what data and technologies they will be looking for," says Gerben Zondag, founder of Immunetune, who will take on the role of Chief Operating Officer. "With the rest of the management team in place, we are confident to move our company and programs forward into the clinic."

Sijme Zeilemaker is joining Immunetune having previously acted as Chief Operating Officer, Director Business Development and Head of Investor Relations at Immunicum AB, a clinical-stage biotech developing cell therapies against cancer, publicly listed at the Nasdaq Stockholm. He joined Immunicum in 2017 as part of a small team and supported the growth of the company through a collaboration with Pfizer and Merck KGaA, three financing rounds, and the merger with DCprime. Before Immunicum, Sijme was Director Business Development at InteRNA Technologies, and held business development roles at to-BBB technologies and 2-BBB Medicines, transitioning these companies from preclinical to clinical-stage.

On August 11, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported financial results for the second quarter of 2021 and provided a business update (Press release, Diffusion Pharmaceuticals, AUG 11, 2021, View Source [SID1234587026]).

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"We met our key milestones for the first half of 2021, completing and announcing topline results from our COVID-19 Trial and the first of our Oxygenation Trials. Our development plan remains on track. With the positive outcomes of the COVID-19 and TCOM studies, as well as our significant capital raise in the first quarter, we believe we have the data and the financial capacity to execute our clinical development strategy for TSC through Phase 2b. For the remainder of 2021, we are focused on the design and execution of the remaining two Oxygenation Trials to further our understanding of TSC’s mechanism of action and potential, as well as the further development of our regulatory and commercial strategy for TSC," said Robert Cobuzzi, Jr. Ph.D., President and CEO of Diffusion.

TSC Q2 Development Update

In May 2021, Diffusion reported final results from its Phase 1b trial (the "COVID-19 Trial") of trans sodium crocetinate ("TSC") in hospitalized COVID-19 patients. Although the study was not designed or powered to evaluate efficacy, the study’s external safety monitoring committee observed that patients receiving the 1.5 mg/kg dose of TSC had improved outcomes in secondary and exploratory endpoints compared to those receiving lower doses. The final results were consistent with topline results previously announced in February 2021, indicating that TSC was safe and well-tolerated when administered on a more frequent dosing regimen than previously tested in a clinical trial setting.

In June 2021, the Company reported a positive trend in oxygenation from its Phase 1b trial (the "TCOM Trial") evaluating TSC using transcutaneous oxygen monitoring ("TCOM"). The TCOM Trial was designed to evaluate the effect of TSC versus placebo on peripheral tissue oxygenation in healthy normal volunteers. Topline results based upon analyses of primary endpoint data indicated, as compared to placebo, a positive dose-response trend in TCOM readings after TSC administration that persisted through the measurement period with no evidence of hyperoxygenation. TSC was also safe and well-tolerated at all doses tested.

TSC Development Plans for 2021 and 2022

The positive trend observed in the TCOM Trial is being used to guide dose selection in the additional Oxygenation Trials planned for the latter part of 2021 – the Altitude Trial, followed by the ILD-DLCO Trial.

Altitude Trial (formerly known as the Induced Hypoxia Trial): This trial will be a double-blind, randomized, placebo-controlled study which will evaluate the effects of TSC on maximal oxygen consumption, or VO2, and partial pressure of blood oxygen, or PaO2, in normal healthy volunteers subjected to incremental levels of physical exertion while exposed to hypoxic and hypobaric conditions (i.e., simulated altitude). The study is designed to evaluate the effect of TSC versus placebo on maximal oxygen consumption and partial pressure of blood oxygen. Diffusion anticipates initiating and completing the Altitude Trial in the fourth quarter of 2021, with topline results available within one to two months of study completion.

ILD – DLCO Trial: This trial will be a double-blind, randomized, placebo-controlled study which will evaluate the effects of TSC on the diffusion of carbon monoxide through the lungs ("DLCO") in patients with previously diagnosed interstitial lung disease ("ILD") who have a baseline DLCO test result that is abnormal. DLCO will act as a surrogate measure of oxygen transfer efficiency, or uptake, from the alveoli of the lungs, through the plasma, and onto hemoglobin within red blood cells. The study will be statistically powered to evaluate the difference in effect of TSC versus placebo on improvement in DLCO as well as in a standard six-minute walk test. Diffusion now anticipates initiating the ILD-DLCO Trial in the late fourth quarter of 2021 and completing the trial in the first quarter of 2022, with topline results available within one to two months of study completion.

Diffusion expects to announce in the fourth quarter of 2021 the initial indication in which TSC will be studied to support the planned pathway for regulatory approval and to initiate a controlled, clinical outcome study evaluating TSC in the chosen indication during the first half of 2022, funded with cash-on-hand.

Operating and Leadership Team Developments

During the second quarter, Diffusion enhanced its operating team with the addition of new employees in the areas of administration, quality assurance, clinical operations, and finance. In addition, in connection with the Diffusion’s annual meeting of stockholders in June 2021, Jane H. Hollingsworth was elected as the new chair of the Company’s board of directors and Diana Lanchoney, M.D. and Eric Francois were newly elected to the board of directors. The Company believes these organizational additions have already had a significant, positive impact on its ability to develop, implement and execute on its corporate strategy and development plans, and position the Company well to build shareholder value through its next stage of growth.

2Q21 Financial Results

Research and development expenses in the second quarter were $2.0 million compared to expenses of $2.2 million in the prior year period. The decrease was attributable to the wind-down of the Company’s clinical trial evaluating TSC in glioblastoma multiforme brain cancer and its COVID-19 Trial completed in February 2021. These decreases were offset by increased headcount and costs related to the TCOM Trial.

General and administrative expenses were $1.8 million during the second quarter of 2021 versus $1.5 million in the comparable quarter last year. The increase compared to the prior year period was primarily attributable to increased salaries, wages, stock-based compensation, and professional fees related to increased headcount and costs associated with the separation of former employees that will not recur in future years.

As of June 30, 2021, Diffusion had cash and cash equivalents of approximately $43.3 million as compared to $18.5 million as of December 31, 2020.